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Stem cell research leads to potential new therapy for rare blood disorder
Date:4/7/2008

A unique partnership between industry and academia has led to human clinical trials of a new drug for a rare class of blood diseases called myeloproliferative disorders (MPD), which are all driven by the same genetic mutation and can evolve into leukemia. In just one year, collaborative discoveries by stem cell researchers from the University of California, San Diego, Dana-Farber Cancer Institute, the Mayo Clinic and a San Diego pharmaceutical company, TargeGen, moved from identification of the most promising drug candidate to clinical trials for a new drug to fight this degenerative blood disorder, which affects more than 100,000 Americans.

A study headed by Catriona H.M. Jamieson, M.D. Ph.D., assistant professor of medicine at the University of California, San Diego and Director for Stem Cell Research at Moores UCSD Cancer Center, found an inhibitor that can stop the over-proliferation of blood cells that results in problems with blood clotting, heart attacks and, in some cases, leukemia. Funded in part by a grant from the California Institute for Regenerative Medicine (CIRM), the study will be published in Cancer Cell on April 8, 2008. A parallel study at Harvard Medical School, headed by D. Gary Gilliland, Ph.D., M.D., yielded similar results which will appear in the same issue of Cancer Cell.

As a clinician, I asked myself who is going to get this disease, and what can we do to stop its progression, instead of waiting until it evolves into a deadly cancer? said Jamieson. This project has been so extraordinary, because a small pharmaceutical company took a big chance on a rare disease.

With major contributions from collaborators Jason Gotlib at Stanford University and Ayalew Tefferi at the Mayo Clinic, the research findings led to development of the inhibitor by TargeGen. That drug is currently being tested in human clinical trials at the UC San Diego School of Medicine, the Mayo Clinic, M.D. Anderson Cancer Center, and th
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Contact: Debra Kain
ddkain@ucsd.edu
619-543-6163
University of California - San Diego
Source:Eurekalert

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